Abeona Therapeutics Inc. (NASDAQ:ABEO)

Abeona Therapeutics Inc (NASDAQ:ABEO) has announced receiving an Orphan Drug Designation (ODD) from the U.S. Federal Drug Administration (FDA) for its EB-101 gene therapy targeting dystrophic epidermolysis bullosa (DEB) patients. This also includes recessive dystrophic epidermolysis bullosa (RDEB), a deadly genetic skin disorder characterized by lesions and skin blisters. Abeona specializes in the development of gene therapies for treatment of life-threatening diseases.

In a statement, Abeona Therapeutics Inc (NASDAQ:ABEO) President & CEO, Timothy J. Miller, Ph.D said Abeona’s main agenda is to develop therapies to address the huge unmet demands from patients suffering from dystrophic epidermolysis bullosa. He added that they highly grateful for the new designation and the recognition of EB-101 by FDA as a rare product which will offer much needed therapeutic benefits to patients suffering from dystrophic types of epidermolysis bullosa among them RDEB. EB-101 is the company’s third gene therapy to be given the Orphan Drug Designation by the FDA that, as Miller states, is an important driver to the company’s program as it offers seven years of therapies for the same indications.

Wounds that appear on patients with RDEB, referred to as “butterfly skin” syndrome, can go unhealed for several months or even years as a result of the inability of the skin to remain attached to the dermis below and can spread over a large part of the body. During the Phase 1/2 clinical study, EB-101 was used to treat non-healing chronic wounds on each patient and examined the process of healing at particular time intervals over years. The clinical trial’s endpoint examined the safety as well as evaluating the healing process and compared the results to control untreated wounds. The secondary endpoint of the trial includes collagen C7 as well as the restoration of anchoring fibrils after three and six months of post-treatment.

EB is a class of deadly genetic skin diseases which leads to growth of erosions and blisters on the skin and spreads over the entire body. The most deadly is the epidermolysis bullosa (RDEB), which is known for open and painful wounds, skin blistering, esophageal strictures, joint contractures, corneal abrasions, pseudosyndactyly and a shortened life span. Patients suffering from RDEB have a shortage of type VII collagen (C7) as a results of gene mutations in the gene COL7A1 which encodes for C7 and the main element of anchoring fibrils, which make the connection between the dermis and the epidermis.

I have no positions in any of the stocks mentioned, and have no plans to initiate any positions within the next 72 hours. All information, including any data, is provided without any guarantees of accuracy.

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About the author: Monica has an undergraduate degree in Accounting and an MBA she earned – with Honors. She has six years of experience in the financial markets and has been an analyst for the past two years.

Abeona Therapeutics Inc. (NASDAQ:ABEO)

Abeona Therapeutics Inc. (NASDAQ:ABEO) has been granted Australian regulatory approval to start a Phase 1/2 study for the ABO-102 gene therapy program targeting Sanfilippo syndrome type A (MPS IIIA) patients. Abeona Therapeutics is a biopharmaceutical company engaged in the development of drugs used in the treatment of life-threatening, rare diseases

In a statement Abeona Therapeutics Inc. (NASDAQ:ABEO)’s President & CEO. Timothy J. Miller, Ph.D, said the company has admitted more patients with MPS IIIA in the trial than any other study has ever admitted in the whole world. The company has also added another clinical site that is expected to speed up the ability to evaluate ABO-102 as a potential Sanfilippo syndrome type A treatment. He added that the company recently announced encouraging clinical data and they expect to admit more patients in Spain and Australia in the near future. He lauded The Sanfilippo Children’s Foundation together with the families for their contribution in producing life-saving treatments.

MPS IIIA is a childhood degenerative disorder and mainly manifests in the form of cognitive decline and progressive neuromotor in addition to systemic complications which eventually lead to a premature death. According to Nicholas Smith, M.D., Ph.D from Women’s and Children’s Hospital the data that was reported has shown high potential to reduce heparan sulphate in the Central Nervous System, reduce liver volume, as well as slow neurocognitive decline.

The ongoing Phase 1/2 clinical study has been given FastTrack Designation, Rare Pediatric Disease Designation, and Orphan Product Designation by the FDA. The Phase trials are aimed at evaluating the safety and efficacy of ABO-102 in MPS IIIA patients. According to the trial design, patients are given one intravenous ABO-102 injection which is the delivery point for the AAV viral vector into the body so as to introduce the corrective copy of the gene which underlies the MPS IIIA disease. Patients are examined at several points in the first six months after the injection for assessment of safety as well as initial signs of biopotency. The world clinical trial has the benefit of a 25-subject MPS III Natural History Study that is being included with several efficacy assessments. They consist of biochemical assays, neurocognitive evaluations, and MRI data produced over one year of follow-up evaluations.

I have no positions in any of the stocks mentioned, and have no plans to initiate any positions within the next 72 hours. All information, including any data, is provided without any guarantees of accuracy.

About the author: Monica Gray has an undergraduate degree in Accounting and an MBA – earned with Honors. She has six years of experience in the financial markets and has been a securities analyst for the past two years.

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